EP3373972C0 - Zusammensetzungen zur behandlung von spinaler muskelatrophie - Google Patents

Zusammensetzungen zur behandlung von spinaler muskelatrophie

Info

Publication number
EP3373972C0
EP3373972C0 EP16794290.3A EP16794290A EP3373972C0 EP 3373972 C0 EP3373972 C0 EP 3373972C0 EP 16794290 A EP16794290 A EP 16794290A EP 3373972 C0 EP3373972 C0 EP 3373972C0
Authority
EP
European Patent Office
Prior art keywords
compositions
treatment
muscle atrophy
spinal muscle
spinal
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
EP16794290.3A
Other languages
English (en)
French (fr)
Other versions
EP3373972B1 (de
EP3373972A1 (de
Inventor
Jochem Alsenz
Olaf Grassmann
Peter Kuehl
Friedrich Metzger
Kathleen Dorothy Mccarthy
Vianna Eduardo Paulo Morawski
Marvin Lloyd Woodhouse
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
F Hoffmann La Roche AG
Original Assignee
F Hoffmann La Roche AG
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=54542056&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=EP3373972(C0) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by F Hoffmann La Roche AG filed Critical F Hoffmann La Roche AG
Priority to EP24156739.5A priority Critical patent/EP4360707A3/de
Publication of EP3373972A1 publication Critical patent/EP3373972A1/de
Application granted granted Critical
Publication of EP3373972B1 publication Critical patent/EP3373972B1/de
Publication of EP3373972C0 publication Critical patent/EP3373972C0/de
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12—Carboxylic acids; Salts or anhydrides thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0087—Galenical forms not covered by A61K9/02 - A61K9/7023
    • A61K9/0095—Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
EP16794290.3A 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie Active EP3373972B1 (de)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP24156739.5A EP4360707A3 (de) 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP15194297 2015-11-12
PCT/EP2016/076905 WO2017080967A1 (en) 2015-11-12 2016-11-08 Compositions for treating spinal muscular atrophy

Related Child Applications (2)

Application Number Title Priority Date Filing Date
EP24156739.5A Division-Into EP4360707A3 (de) 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie
EP24156739.5A Division EP4360707A3 (de) 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie

Publications (3)

Publication Number Publication Date
EP3373972A1 EP3373972A1 (de) 2018-09-19
EP3373972B1 EP3373972B1 (de) 2024-03-20
EP3373972C0 true EP3373972C0 (de) 2024-03-20

Family

ID=54542056

Family Applications (2)

Application Number Title Priority Date Filing Date
EP24156739.5A Pending EP4360707A3 (de) 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie
EP16794290.3A Active EP3373972B1 (de) 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie

Family Applications Before (1)

Application Number Title Priority Date Filing Date
EP24156739.5A Pending EP4360707A3 (de) 2015-11-12 2016-11-08 Zusammensetzungen zur behandlung von spinaler muskelatrophie

Country Status (15)

Country Link
US (3) US20180289712A1 (de)
EP (2) EP4360707A3 (de)
JP (1) JP6659841B2 (de)
KR (1) KR102162062B1 (de)
CN (1) CN108289959B (de)
AR (1) AR106717A1 (de)
AU (1) AU2016353961B2 (de)
BR (1) BR112018009281B1 (de)
CA (1) CA3002494C (de)
EA (1) EA036399B1 (de)
ES (1) ES2988532T3 (de)
IL (1) IL258497B (de)
MX (1) MX384868B (de)
PL (1) PL3373972T3 (de)
WO (1) WO2017080967A1 (de)

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WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
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WO2020231977A1 (en) 2019-05-13 2020-11-19 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
CN113966219A (zh) 2019-06-12 2022-01-21 豪夫迈·罗氏有限公司 Sma的新治疗方法
CN113993452A (zh) * 2019-06-19 2022-01-28 豪夫迈·罗氏有限公司 数字生物标志物
JP2022537266A (ja) * 2019-06-19 2022-08-25 エフ．ホフマン－ラ ロシュ アーゲー デジタルバイオマーカー
JP7687968B2 (ja) 2019-06-19 2025-06-03 エフ． ホフマン－ラ ロシュ アーゲー デジタルバイオマーカー
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Also Published As

Publication number Publication date
US11938136B2 (en) 2024-03-26
IL258497A (en) 2018-06-28
BR112018009281A2 (pt) 2018-11-06
KR102162062B1 (ko) 2020-10-07
BR112018009281A8 (pt) 2019-02-26
EP3373972B1 (de) 2024-03-20
CN108289959A (zh) 2018-07-17
AU2016353961B2 (en) 2019-08-29
CA3002494A1 (en) 2017-05-18
HK1256089A1 (zh) 2019-09-13
US20240293410A1 (en) 2024-09-05
EP4360707A3 (de) 2024-08-07
EP3373972A1 (de) 2018-09-19
BR112018009281B1 (pt) 2023-10-31
CN108289959B (zh) 2021-07-16
EP4360707A2 (de) 2024-05-01
CA3002494C (en) 2021-01-12
AR106717A1 (es) 2018-02-14
AU2016353961A1 (en) 2018-04-26
WO2017080967A1 (en) 2017-05-18
KR20180080317A (ko) 2018-07-11
IL258497B (en) 2021-07-29
EA036399B1 (ru) 2020-11-06
US20180289712A1 (en) 2018-10-11
MX384868B (es) 2025-03-14
MX2018005890A (es) 2018-08-15
JP2018533605A (ja) 2018-11-15
EA201891103A1 (ru) 2018-11-30
ES2988532T3 (es) 2024-11-20
US20200323856A1 (en) 2020-10-15
JP6659841B2 (ja) 2020-03-04
PL3373972T3 (pl) 2024-06-17

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